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Trial record 17 of 180 for:    "Mitochondrial Diseases" OR "mitochondrial neurogastrointestinal encephalopathy disease"

Open-Label, Dose-Escalating Study Assessing Safety, Tolerability, Efficacy, of RP103 in Mitochondrial Disease (MITO-001)

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ClinicalTrials.gov Identifier: NCT02023866
Recruitment Status : Completed
First Posted : December 30, 2013
Results First Posted : November 13, 2017
Last Update Posted : November 13, 2017
Sponsor:
Information provided by (Responsible Party):
Horizon Pharma USA, Inc.

Study Type: Interventional
Study Design: Intervention Model: Single Group Assignment;   Masking: None (Open Label);   Primary Purpose: Treatment
Condition: Inherited Mitochondrial Disease, Including Leigh Syndrome
Intervention: Drug: Cysteamine Bitartrate

  Participant Flow

Recruitment Details
Key information relevant to the recruitment process for the overall study, such as dates of the recruitment period and locations
No text entered.

Pre-Assignment Details
Significant events and approaches for the overall study following participant enrollment, but prior to group assignment
No text entered.

Reporting Groups
  Description
Cysteamine Bitartrate Delayed-release Cysteamine bitartrate delayed-release capsules were administered twice daily following a dose-escalation design with a progressive weekly dose increase over the first 6 weeks. The starting dose was 0.2 g/m²/day, up to a maximum dose of 1.3 g/m²/day. Participants remained on their highest tolerated dose until Week 24.

Participant Flow:   Overall Study
    Cysteamine Bitartrate Delayed-release
STARTED   36 
COMPLETED   30 
NOT COMPLETED   6 
Non-compliance                2 
Adverse Event                3 
Withdrawal by Subject                1 



  Baseline Characteristics

Population Description
Explanation of how the number of participants for analysis was determined. Includes whether analysis was per protocol, intention to treat, or another method. Also provides relevant details such as imputation technique, as appropriate.
No text entered.

Reporting Groups
  Description
Cysteamine Bitartrate Delayed-release Cysteamine bitartrate delayed-release capsules were administered twice daily following a dose-escalation design with a progressive weekly dose increase over the first 6 weeks. The starting dose was 0.2 g/m²/day, up to a maximum dose of 1.3 g/m²/day. Participants remained on their highest tolerated dose until Week 24.

Baseline Measures
   Cysteamine Bitartrate Delayed-release 
Overall Participants Analyzed 
[Units: Participants]
 36 
Age 
[Units: Years]
Mean (Standard Deviation)
 9.3  (4.8) 
Sex: Female, Male 
[Units: Participants]
Count of Participants
 
Female      17  47.2% 
Male      19  52.8% 
Ethnicity (NIH/OMB) 
[Units: Participants]
Count of Participants
 
Hispanic or Latino      5  13.9% 
Not Hispanic or Latino      31  86.1% 
Unknown or Not Reported      0   0.0% 
Race/Ethnicity, Customized 
[Units: Participants]
Count of Participants
 
American Indian or Alaska Native      3   8.3% 
Asian      0   0.0% 
Black of African American      0   0.0% 
Native Hawaiian or other Pacific Islander      0   0.0% 
White      28  77.8% 
Other      2   5.6% 
Multiple      3   8.3% 
Mitochondrial Disease Subtype [1] 
[Units: Participants]
Count of Participants
 
Leigh Syndrome      9  25.0% 
POLG-related disorders      7  19.4% 
MELAS      6  16.7% 
MERFF      4  11.1% 
Others      4  11.1% 
LHON      3   8.3% 
NUBPL Related Encephalopathy      2   5.6% 
Kearns-Sayre syndrome      1   2.8% 
[1] LHON = leber hereditary optic neuropathy; MELAS = mitochondrial encephalomyopathy, lactic acidosis, and strokelike syndrome MERFF = myoclonic epilepsy and ragged-red fibers; NUBPL = Nucleotide Binding Protein-Like; POLG = Polymerase (DNA Directed), Gamma.


  Outcome Measures

1.  Primary:   Change From Baseline in Newcastle Paediatric Mitochondrial Disease Scale (NPMDS) Sections I-IV   [ Time Frame: Baseline through Week 24 ]

2.  Secondary:   Change From Baseline in Glutathione   [ Time Frame: Baseline and Weeks 4, 8, 12, 16, 20, 24 ]

3.  Secondary:   Change From Baseline in Glutathione Disulfide   [ Time Frame: Baseline and Weeks 4, 8, 12, 16, 20, 24 ]

4.  Secondary:   Change From Baseline in Lactic Acid   [ Time Frame: Baseline and Weeks 4, 8, 12, 16, 20, 24 ]

5.  Secondary:   Change From Baseline in 6 Minute Walk Test   [ Time Frame: Baseline and Weeks 4, 8, 12, 16, 20, 24 ]

6.  Secondary:   Change From Baseline in Jamar Dynamometer Hand Strength   [ Time Frame: Baseline and Weeks 4, 8, 12, 16, 20, 24 ]

7.  Secondary:   Change From Baseline in Barry-Albright Dystonia Scale Total Score   [ Time Frame: Baseline and Weeks 4, 8, 12, 16, 20, 24 ]

8.  Secondary:   Change From Baseline in Friedreich Ataxia Rating Scale   [ Time Frame: Baseline and Weeks 4, 8, 12, 16, 20, 24 ]

9.  Secondary:   Change From Baseline in Gross Motor Function   [ Time Frame: Baseline and Weeks 4, 8, 12, 16, 20, 24 ]

10.  Secondary:   Change From Baseline in Modified Lansky Play Performance Scale   [ Time Frame: Baseline and Weeks 4, 8, 12, 16, 20, 24 ]


  Serious Adverse Events


  Other Adverse Events


  Limitations and Caveats

Limitations of the study, such as early termination leading to small numbers of participants analyzed and technical problems with measurement leading to unreliable or uninterpretable data
No text entered.


  More Information

Certain Agreements:  
Principal Investigators are NOT employed by the organization sponsoring the study.
There IS an agreement between Principal Investigators and the Sponsor (or its agents) that restricts the PI's rights to discuss or publish trial results after the trial is completed.
The agreement is:
unchecked The only disclosure restriction on the PI is that the sponsor can review results communications prior to public release and can embargo communications regarding trial results for a period that is less than or equal to 60 days. The sponsor cannot require changes to the communication and cannot extend the embargo.
unchecked The only disclosure restriction on the PI is that the sponsor can review results communications prior to public release and can embargo communications regarding trial results for a period that is more than 60 days but less than or equal to 180 days. The sponsor cannot require changes to the communication and cannot extend the embargo.


Results Point of Contact:  
Name/Title: Evelyn Olson, Director
Organization: Horizon Pharma USA, Inc.
phone: 224- 383-3000
e-mail: clinicaltrials@horizonpharma.com


Publications:

Responsible Party: Horizon Pharma USA, Inc.
ClinicalTrials.gov Identifier: NCT02023866     History of Changes
Other Study ID Numbers: RP103-MITO-001
First Submitted: December 17, 2013
First Posted: December 30, 2013
Results First Submitted: October 13, 2017
Results First Posted: November 13, 2017
Last Update Posted: November 13, 2017